Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing.
about
Genome-editing Technologies for Gene and Cell TherapyApplications of CRISPR/Cas9 for Gene Editing in Hereditary Movement DisordersAblation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis.N-terminal Huntingtin Knock-In Mice: Implications of Removing the N-terminal Region of Huntingtin for Therapy.The pathogenic exon 1 HTT protein is produced by incomplete splicing in Huntington's disease patientsThiol-disulfide Oxidoreductases TRX1 and TMX3 Decrease Neuronal Atrophy in a Lentiviral Mouse Model of Huntington's DiseaseIdentification of Genetic Factors that Modify Clinical Onset of Huntington's Disease.Sequence-Level Analysis of the Major European Huntington Disease Haplotype.Linking white matter and deep gray matter alterations in premanifest Huntington diseaseSafety of Striatal Infusion of siRNA in a Transgenic Huntington's Disease Mouse Model.Induced Pluripotent Stem Cells in Huntington's Disease: Disease Modeling and the Potential for Cell-Based Therapy.AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.Bone Marrow-Derived Cells as a Therapeutic Approach to Optic Nerve Diseases.Destination Brain: the Past, Present, and Future of Therapeutic Gene Delivery.Molecular Pathophysiology of Fragile X-Associated Tremor/Ataxia Syndrome and Perspectives for Drug Development.Investigational agents for the management of Huntington's disease.Huntington's Disease-Update on Treatments.Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease.Cellular Analysis of Silencing the Huntington's Disease Gene Using AAV9 Mediated Delivery of Artificial Micro RNA into the Striatum of Q140/Q140 Mice.Effects of Exogenous NUB1 Expression in the Striatum of HDQ175/Q7 Mice.Post-translational modifications clustering within proteolytic domains decrease mutant huntingtin toxicity.Validation of Ultrasensitive Mutant Huntingtin Detection in Human Cerebrospinal Fluid by Single Molecule Counting Immunoassay.Correction of Huntington's Disease Phenotype by Genistein-Induced Autophagy in the Cellular Model.The potential of antisense oligonucleotide therapies for inherited childhood lung diseases.Striatal Mutant Huntingtin Protein Levels Decline with Age in Homozygous Huntington's Disease Knock-In Mouse Models.A Fresh Look at Huntingtin mRNA Processing in Huntington's Disease.Quantification of Total and Mutant Huntingtin Protein Levels in Biospecimens Using a Novel alphaLISA Assay
P2860
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P2860
Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing.
description
2014 nî lūn-bûn
@nan
2014年の論文
@ja
2014年学术文章
@wuu
2014年学术文章
@zh-cn
2014年学术文章
@zh-hans
2014年学术文章
@zh-my
2014年学术文章
@zh-sg
2014年學術文章
@yue
2014年學術文章
@zh
2014年學術文章
@zh-hant
name
Huntingtin-lowering strategies ...... small RNAs, and gene editing.
@en
type
label
Huntingtin-lowering strategies ...... small RNAs, and gene editing.
@en
prefLabel
Huntingtin-lowering strategies ...... small RNAs, and gene editing.
@en
P2860
P356
P1433
P1476
Huntingtin-lowering strategies ...... small RNAs, and gene editing.
@en
P2093
Marian DiFiglia
Neil Aronin
P2860
P304
P356
10.1002/MDS.26020
P407
P577
2014-08-27T00:00:00Z